A Phase 1 interventional study of Topotecan in Proliferative Vitreoretinopathy, Proliferative Vitreo-Retinopathy and Rhegmatogenous Retinal Detachment, sponsored by Massachusetts Eye and Ear Infirmary. Not yet recruiting. Open to participants aged 18 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-10-16.
Sponsored by Massachusetts Eye and Ear Infirmary · Phase 1, Interventional, and Treatment
The goal of this clinical trial is to evaluate the safety and efficacy of intravitreal topotecan for the treatment of patients with rhegmatogenous retinal detachment due to proliferative vitreoretinopathy (PVR) or resulting from an open globe injury, and compare the outcomes to those who do no receive intravitreal topotecan. The main objectives it aims to achieve are:
Post-consent, participants will:
Researchers will compare participants who receive intravitreal topotecan for PVR to those who do not to see if there is a significant variability in recurrence of retinal detachment, rate of retinal reattachment and PVR grade 6 months after surgery.
Rhegmatogenous retinal detachment (RRD) is an acute, sight-threatening condition that occurs after separation of the neurosensory retina from the underlying retinal pigment epithelium (RPE) due to the presence of a retinal break. Proliferative vitreoretinopathy (PVR) represents growth of ectopic fibrocellular growth on the surface of, within and underneath the retina. PVR is hypothesized to occur secondary to the migration of RPE cells to the peri-retina, leading to a mesenchymal transition into contractile myofibroblasts. PVR affects 5-10% of RRDs and is the most common cause of surgical failure in RRD.
Given that PVR involves a pro-inflammatory and pro-fibrotic cellular response, adjuvants such as corticosteroids and antimetabolites such as 5-fluorouracil have been attempted in the treatment of this condition. Overall, the efficacy results of these treatments have been mixed, and no standard of care adjuvant therapy has emerged.
Topotecan is a chemotherapeutic agent that acts as a topoisomerase inhibitor. It is most commonly administered intravitreally for patients with refractory or recurrent vitreous seeds from retinoblastoma, and shows high efficacy in this setting. At the same time, intravitreal topotecan administered at doses of 5-30µg has been associated with no ocular or systemic complications or adverse electroretinogram changes. To the best of available knowledge, topotecan has not been administered for the treatment of proliferative vitreoretinopathy. Topotecan has anti-inflammatory, anti-proliferative and anti-fibrotic activity that is hypothesized to exhibit high efficacy for the treatment of PVR. In this study, the efficacy and safety of intravitreal topotecan for the treatment of PVR will be investigated.
43 studies on the registry are indexed under Vitreoretinopathy, Proliferative; 11 are open to participants now.
This study's planned enrollment of 50 is below the median of 64 across 30 interventional studies indexed under Vitreoretinopathy, Proliferative.
Browse Vitreoretinopathy, Proliferative studies →Massachusetts Eye and Ear Infirmary is the lead sponsor of 100 studies on the registry; 23 are open to participants now.
Of its 13 completed or terminated interventional studies of FDA-regulated products, 7 (54%) have results posted.
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Exclusion Criteria:
These are the patients who will be receiving intravitreally 20 micrograms of topotecan in a 1cc syringe during surgery, at the post-operative day 7 and at the post-operative day 28.
Drug: Topotecan
These patients will not be receiving any intervention.
20 micrograms of intravitreal topotecan given in a 1 cc tuberculin syringe at a concentration of 20 mcg/20mcL.
Also known as: Hycamtin
Recurrence of rhegmatogenous retinal detachment secondary to PVR
Investigators will be evaluating whether participant develop a recurrent retinal detachment throughout their follow up after their initial surgery.
Time frame: 6 months after initial surgery, or last follow-up visit available
Best corrected visual acuity (BCVA)
Investigators will be measuring the BCVA of participants throughout their follow up and see if they notice any change from surgery time to final follow up
Time frame: at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168
Variation of PVR grade
Investigators will be doing a dilated fundus exam and taking fundus photographies for all participants throughout their follow up time to see if the grade of the PVR changes over time. PVR grade goes from grade A to grade C-Anterior to equator/ C-Posterior to equator (grade C being worse than grade A)
Time frame: at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168
Retinal reattachment rate at month 6 or last follow up
Investigators will be doing a dilated fundus exam and taking fundus photographies for all participants throughout their follow up time to see if the retinal reattaches (whether completely or partially) after surgery
Time frame: at post-operative day 168 (or last follow-up visit available if the participant did not show up at post-operative day 168)
Recurrence of rhegmatogenous retinal detachment due to any cause
Investigators will be measuring the BCVA of participants throughout their follow up and see if they notice any change from surgery time to final follow up
Time frame: at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168
Number of participants and type of intraoperative or postoperative complications
Any adverse event will be noted during surgery, and at each follow up visit for each participant if applicable.
Time frame: at time of surgery, and each follow up visit (post-operative day1, 7, 28, 56, 84, 126, 168)
Optical coherence tomography (OCT) of the macula
an OCT of the macula will be taken to evaluate for any retinal changes throughout the study
Time frame: at the pre-operative assessment, and post-operative day1, 7, 28, 56, 84, 126, 168
No study locations are listed for this record.
Plan to share: No
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Topotecan
Vitreoretinopathy, Proliferative →
Massachusetts Eye and Ear Infirmary →